Emerging treatments

COX-2

The enzyme COX-2 has been shown to be over-expressed in both the intestinal hamartomas and cancers associated with PJS.[8][9] Limited evidence has demonstrated a benefit from the use of celecoxib, a COX-2 inhibitor, in decreasing polyp burden in experimental animals and humans with PJS and appears to be a possible target for chemo-prevention research.[42] To date, there is insufficient evidence to recommend pharmacological agents as a treatment or for chemoprevention in PJS.[13][14]

mTOR inhibitors

LKB1, the serine-threonine kinase encoded by STK11, modulates PI3-kinase signalling, which is a key regulator of cell growth and survival. The mammalian target of rapamycin, mTOR, is an important downstream regulator of PI3-kinase signalling. Studies of Lkb1 ± mice have shown that treatment with rapamycin reduced the number of intestinal hamartomatous polyps.[43][44] Rapamycin and everolimus have been studied, but no conclusions can be drawn.[45][46]

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